[P19] 代謝性疾患・神経変性疾患

[P19-07] Real-World Assessment of Onasemnogene Abeparvovec in Patients with SMA: Interim Report of the RESTORE Registry in Japan

齋藤 加代子¹, Benguerba Kamal², Tsuchida Ken³, Yazawa Kazushige³, Tsumiyama Isao³, Kayama Hiromitsu³, Reyna Sandra P.⁴, Khan Farid⁴, Finkel Richard S.⁵ (1.Tokyo Women′s Medical University, 2.Novartis Pharma AG, CH-4056 Basel, Switzerland, 3.Novartis Pharma K.K., Tokyo, Japan, 4.Novartis Gene Therapies, Inc., Bannockburn, IL, USA, 5.Center for Experimental Neurotherapeutics, St. Jude Children′s Research Hospital, Memphis, TN, USA)

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